Yesterday, the 21st Century Cures bill was unanimously passed by the House Energy and Commerce Committee. 21st Century Cures would overhaul how the Food and Drug Administration evaluates new medical products and provide $10 billion in mandatory funding for the National Institutes of Health. Passage by the Committee is a significant milestone for this important […]
21st Century Cures Unanimously Passes Committee Read More »
Much as families in our community often work together to start a chapter, organize an event, or raise awareness of SMA, researchers must work together in order to find the fastest and most effective path to a cure for SMA. In addition to providing funding for ongoing SMA research, Cure SMA provides several opportunities for
Collaborative Ways Cure SMA is Driving Research Results Read More »
In a recent update on the 21st Century Cures Initiative, we shared how the SMA community has helped inspire this legistlation. Chairman Fred Upton has been a guest at our Hope on the Hill Congressional Dinner. He has also formed a strong relationship with Brooke and Brielle Kennedy, two sisters from his home state of
Meet the SMA Family that Helped Inspire 21st Century Cures Read More »
Cytokinetics, in partnership with Astellas, has officially announced that recruitment will begin in the second half of 2015 for a Phase 2 clinical trial testing CK-2127107, a muscle activator. This randomized, double-blind study is expected to enroll 75 patients with SMA type II, III, or IV. Patients will be dosed for four weeks, and will
Cytokinetics and Astellas Release Further Details on Upcoming Phase 2 Trial Read More »
Cure SMA has become a participating organization of The Research Acceleration and Innovation Network (TRAIN), a program of FasterCures, bringing the total number of venture philanthropies in TRAIN to 80. FasterCures established TRAIN in 2005 to connect disruptive innovators in the disease research space with the vital resources, tools and relationships to catalyze development of
Cure SMA Joins The Research Acceleration and Innovation Network Read More »
The SMA drug pipeline has grown dramatically in just over a decade. Of the 17 programs in the pipeline, seven of these are in clinical trials, and two of those are in Phase 3 clinical trials. Reaching and achieving FDA approval is a major hurdle in our path to a treatment and cure. And, consistent
Update on SMA-FDA Interactions Read More »
The Family Friendly Research Poster Session is one of the most anticipated events of our Annual SMA Conference. Each year, Cure SMA invites researchers from the SMA Researcher Meeting, which is held in parallel with the family conference, to present their findings to families in way that is accessible to them. During the event, families
2015 Family Friendly Poster Session Presenters Announced Read More »
F. Hoffman-La Roche, PTC and the SMA Foundation recently provided an update on the clinical development of RG7800, an investigational SMN2 splicing modifier that is being studied as a potential treatment for spinal muscular atrophy (SMA): “In November 2014, Roche initiated the Moonfish trial (Phase 1b/2a) based on supportive results from a trial in healthy
Update on Clinical Development of RG7800 Read More »
Over the past few months, Cure SMA has announced a total of $1.85 million in new research funding. Our model is designed to fund both a breadth and a depth of projects, in order to continuing building up both a breadth and depth of programs in the drug pipeline. This includes funding for: Basic research,
Cure SMA Funds $1.85 Million in New Research Grants in 2014-2015 Read More »
Cure SMA, the leading organization dedicated to the treatment and cure of spinal muscular atrophy (SMA), recently provided an additional $445,000 grant toward a gene therapy for SMA at Nationwide Children’s Hospital. Brian Kaspar, PhD, principal investigator in the Center for Gene Therapy at Nationwide Children’s, and his team have made promising strides toward therapies
